Andrew Kuykendall, MD, Clinical Researcher at Moffitt Cancer Center, discusses the role of rusfertide in decreasing phlebotomies in patients with polycythemia vera (PV).
PV is a condition characterized by an increased number of red blood cells. Affected people may also have excess white blood cells and platelets. The excess cell levels lead to thicker blood and increased risk for blood clots. PV occurs more frequently in men than it does in women. The condition has been associated with genetic changes in the JAK2 and TET2 genes.
Rusfertide is a first-in-class hepcidin mimetic peptide observed to reduce the need for phlebotomy and control hematocrit levels by regulating iron distribution and restricting iron availability for red blood cell production in patients with PV.
Dr. Kuykendall explains how phlebotomies have become so ingrained in the treatment of PV, that the burden it places on patients has not been adequately addressed. However, a recent focus on patient burden is bringing the impact of this treatment strategy to light. Frequent doctor’s office and hospital visits to get labs checked and then get phlebotomies every two to three weeks greatly limit patient’s freedom and autonomy.
While these phlebotomies may help the overproduction of red blood cells in these patients, they do not address the iron deficiency and consequent fatigue, brain fog, and other disabling symptoms that affect patient’s daily lives. Dr. Kuykendall explains how the development of rusfertide was based on mimicking a high-hepcidin state to increase iron levels, control hematocrit, and decrease the need for phlebotomy by reestablishing balance of iron regulation, thus improving fatigue and disease-related symptoms. Additionally, rusfertide may be added to patients on cytoreductive therapy doses that are higher than are comfortably tolerated, to help more optimally dose patients.
Unmet needs still exist for patients with PV, including the development of therapies that address the underlying JAK2 mutation that causes disease, as well as those that prevent progression.
Finally, Dr. Kuykendall highlights that rusfertide was developed by listening to patients and that it should be used in practice the same way, stressing the importance of focusing on how the disease affects them and how this treatment could fit into patients’ lives.
To learn more about PV and other rare hematologic conditions, visit https://checkrare.com/diseases/hematologic-disorders/
